Background Many medicines prescribed for children have not been formally studied. Therefore, most of them are not labelled to be used in paediatric patients (PP). The Regulation (EC) No 1901/2006 on medicinal products for paediatric use sets up several requirements, rewards and incentives to promote medicinal products being researched, developed and authorised covering the therapeutic needs of children.
Purpose To evaluate the prescription profile of antineoplastic agents in PP in two relevant hospitals in paediatrics that belong to a Regional Health Service.
Material and methods Descriptive study of antineoplasic drug prescription in the PP in the Oncology and Haematology Departments during 2017. Data collected: age and drug (brand/generic name and active substance). Indications and use conditions for the prescribed drugs were evaluated according to the EMA summary of product characteristics They were classified as approved, unlicensed or off-label. Unlicensed drug use was defined as the use of a non-marketed drug. Off-label drug use was defined as the use of a drug in unapproved conditions.
Results A total of 220 children were included (average age: 7.77 years). Six-hundred and forty-seven prescriptions (involving 52 different active substances (AS)) were evaluated. 68.32% of all prescriptions were approved drugs (26 different AS), 12.98% were unlicensed drugs (13 different AS) and 18.70% were off-label drugs (18 different AS). One-hundred and eighty-one children received at least one approved drug, 73 at least one unlicensed drug and 95 at least one off-label drug. Unlicensed and off-label drug use in younger children was higher: 51.24% in 0–4 year old children received at least one unlicensed or off-label drug against 45.57% of 12–17 year old children. Most commonly prescribed approved drugs were: vincristine (12.52%), cyclophosphamide (8.81%) and cytarabine (8.50%). Most commonly prescribed unlicensed drugs were: mercaptopurine oral solution (3.71%), dactynomicin (2.63%) and pegaspargase (2.16%). Most commonly prescribed off-label drugs were: carboplatin (3.71%), bevacizumab (2.78%) and ifosfamide (2.32%).
Conclusion Despite Regulation (EC) No 1901/2006, the results of our study show that around two-thirds of the children received at least one unlicensed or off-label antineoplasic agent.
Proper clinical studies demonstrating and supporting the use of effective and safe drugs on PP are required.
References and/or acknowledgements No conflict of interest.